RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
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Ultragenyx has submitted a Biologics License Application (BLA) to the FDA for UX111, a gene therapy targeting Sanfilippo syndrome type A, seeking accelerated approval in the US.
December 20, 2024 | 5:45 pm
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Ultragenyx has submitted a BLA to the FDA for UX111, a gene therapy for Sanfilippo syndrome type A, which could lead to accelerated approval and potentially boost the company's market position.
The submission of a BLA for UX111 indicates progress in Ultragenyx's product pipeline, which is crucial for biotech companies. Accelerated approval could lead to earlier market entry, potentially increasing revenue and enhancing investor sentiment.
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